Josh Mandel-Brehm, Camp4 Therapeutics CEO (Camp4)

Go­ing af­ter the phe­nom­e­non of 're­gR­NA,' Rick Young's Camp4 se­cures a launch round to push two pro­grams for­ward

You’ve heard of mR­NA, large­ly thanks to the Covid-19 vac­cines de­vel­oped by Pfiz­er/BioN­Tech and Mod­er­na. If you watch the R&D space close­ly, you’ve al­so prob­a­bly heard of oR­NA and eR­NA.

Rick Young

Now, a new biotech out of Rick Young’s White­head In­sti­tute lab at MIT wants to in­tro­duce you to reg­u­la­to­ry RNA, or re­gR­NA.

The com­pa­ny is Camp4 Ther­a­peu­tics, and it’s locked down its first fundraise with a $45 mil­lion Se­ries A on Tues­day. Led by CEO Josh Man­del-Brehm, Camp4 wants to cre­ate oligonu­cleotide-based med­i­cines that con­trol pro­tein ex­pres­sion to help reg­u­late any kind of ge­net­ic dis­ease.

It’s an ef­fort that got start­ed back in 2016, Man­del-Brehm told End­points News, when Young teamed up with sci­en­tif­ic co-founder Leonard Zon at Boston Chil­dren’s Hos­pi­tal. The core of their re­search cen­tered on un­der­stand­ing how cells make de­ci­sions to con­trol their genes, and they be­gan map­ping out cells them­selves.

To do so re­quired hu­man cells, a “whole ar­ray” of next-gen­er­a­tion se­quenc­ing tech­niques and a part­ner to ap­ply a steady AI hand in gen­er­at­ing da­ta, Man­del-Brehm said. Young and Zon be­came able to con­trol drug sig­nal­ing for non-can­cer in­di­ca­tions where they could change a dis­ease’s un­der­ly­ing gene ex­pres­sion in or­der to spur a ther­a­peu­tic ben­e­fit.

They ran in­to a prob­lem fo­cus­ing orig­i­nal­ly on small mol­e­cules — the drugs worked too well, the CEO told End­points, al­ter­ing the genes to a de­gree much more than they would have liked.

“They were ac­ti­vat­ing too many genes. It was too non­spe­cif­ic for us,” Man­del-Brehm said. “It was a good prob­lem to have, but we asked our­selves, ‘How do we get more spe­cif­ic?’”

Young then reeval­u­at­ed the way he thought about tran­scrip­tion, lead­ing him to RNA re­search. While look­ing in­to the mat­ter, he turned to a class of RNA mol­e­cules that don’t con­tain a core pro­tein and are on­ly tran­scribed. These RNAs had been known to re­searchers be­fore, but few knew of their func­tion be­cause they’d get se­quenced out with rel­a­tive fre­quen­cy.

Rather than con­tin­ue ig­nor­ing these reg­u­la­to­ry RNAs, the founders be­gan try­ing to har­ness them for drug­gable pur­pos­es. The cel­lu­lar maps they’d cre­at­ed for the ear­li­er ef­fort proved piv­otal here, Man­del-Brehm said, be­cause re­searchers can’t iden­ti­fy or char­ac­ter­ize the re­gR­NAs with­out them.

All of a sud­den, Camp4’s plat­form shift­ed to tar­gets. Young and Zon knew they were on­to some­thing and had one last puz­zle piece to solve: how to best at­tack the dis­eases they want­ed to treat, giv­en that small mol­e­cules didn’t suit them. They set­tled on oligonu­cleotides due to the vast amount of pre­vi­ous re­search that’s gone in­to the field.

Over the last 15 months, Camp4 has built out its pre­clin­i­cal pipeline and IP, but they need­ed cash to take things fur­ther. They got a boost in ear­ly 2020 when they signed a li­cens­ing deal with Bio­gen to col­lab­o­rate on mi­croglial cells re­lat­ed to the CNS. But for their own pipeline, the Cam­bridge, MA-based biotech is aim­ing to ini­tial­ly tar­get dis­eases for the liv­er and CNS, hop­ing to file INDs by the end of next year for pro­grams tar­get­ing OTC de­fi­cien­cy and Dravet syn­drome.

Man­del-Brehm has big­ger plans than that though, as he con­tin­ues chart­ing Camp4’s path for­ward. The biotech has about 25 to 30 CNS dis­or­ders they’re look­ing at, and the CEO is aim­ing to be at the fore­front of this new area of bi­ol­o­gy.

“The punch­line is no one is able to go af­ter up­reg­u­la­tion dis­eases, but we’re tak­ing ad­van­tage,” he said. “Peo­ple have al­ways thought of down­reg­u­la­tion, but up is a whole dif­fer­ent cat­e­go­ry. Our sense is it’s go­ing to be a very hot field.”

5AM Ven­tures and North­pond Ven­tures led Tues­day’s fi­nanc­ing along­side ex­ist­ing in­vestors An­dreessen Horowitz, Po­laris Part­ners and The Kraft Group.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a rather narrow market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Time for round 2: Il­lu­mi­na-backed VC snags $325M for its next fund

Illumina Ventures closed off its second investment fund with a total commitment of $325 million, offering fresh fuel to back a slate of startups that have already included a smorgasbord of companies, covering everything from diagnostics to biotech drug development and genomics.

Fund II brings the total investment under Illumina Ventures’ oversight to $560 million, which has been focused on early-stage companies. And it has a transatlantic portfolio that includes SQZ, Twist and Encoded Therapeutics.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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